The Cystic Fibrosis Foundation announced that it making an additional investment of up to $8.5 million to support a Phase 2 trial of SpliSense’s nebulized SPL84 antisense oligonucleotide in patients with cystic fibrosis associated with the 3849+10 kb C-to-T mutation. SPL84 has received Orphan Drug designation from both the EMA and the FDA. According to SpliSense, the FDA has cleared an IND for the study based on positive results from a Phase 1a trial of SPL84.
SpliSense CEO Gili Hart commented, “We are excited to initiate the Phase 2 study for SPL84 in CF with the support of the CF Foundation and other existing investors. Although currently available CF therapies have managed to significantly increase life expectancy over the past few decades, there is still a significant unmet medical need for disease modifying treatments. Our lead product, SPL84, has been shown to fully restore CFTR activity in the CF gold standard pharmacological model.”
Hart continued, “With the recent FDA IND clearance, we hope to be able to deliver, a life-changing treatment for people with CF carrying the 3849+10 Kb C->T mutation and expand our unique technology to additional CF mutations where there is a significant unmet need. In addition, SPL84 serves as proof-of-concept for our platform for the treatment of additional pulmonary diseases, including muco-obstructive diseases and IPF, which are expected to enter the clinic over the next 12 months”
Foundation Executive VP and Chief Scientific Officer Steven M. Rowe said, “We continue to pursue diverse strategies to develop potential treatments for people with CF who can’t benefit from existing modulator therapies. Information from this study is key to advancing those efforts with a novel technology and will also provide valuable insight into the development of therapies for people with rare mutations.”
Read the Cystic Fibrosis Foundation press release.
Read the SpliSense press release.
